Executive Summary

‘Believe it or not, we don’t have as many disease-specific guidances as you would suspect on the new drug side,’ Commissioner Gottlieb says.

The US FDA is planning a guidance on Alzheimer’s drug development, part of a broader reform of the Office of New Drugs (OND).

The guidance-writing initiative is intended to articulate modern evidentiary and clinical trial design expectations in specific disease areas where the science has evolved, FDA Commissioner Scott Gottlieb said Sept. 13 at the Blueprint for Breakthroughs meeting sponsored by Friends of Cancer Research (FOCR) and Alexandria Real Estate Equities.

The goal, Gottlieb said, is to “create more incentives for people to try to attack these unmet medical needs because we have clear direction that they can shoot against.”

Gottlieb first announced the guidance initiative, including at target of at least 10 disease-specific documents, in a speech at the Regulatory Affairs Professionals Society (RAPS) annual conference two days earlier. (Also see “FDA’s Gottlieb Pushing ‘Seamless’ Clinical Trials For Faster Development” – Pink Sheet, 11 Sep, 2017.)

Asked after the initiative during the FOCR/Alexandria meeting, Gottlieb said, “Believe it or not, we don’t have as many disease-specific guidances as you would suspect on the new drug side.”

The OND reform effort that includes the guidance-writing is being spearheaded by Center for Drug Evaluation and Research Director Janet Woodcock, Gottlieb noted. Woodcock took the helm of OND on an acting basis following the retirement of the office’s long-time director, John Jenkins, in January. (Also see “‘Brilliant’ and ‘Steady’ Jenkins Retiring From US FDA’s Office of New Drugs” – Pink Sheet, 5 Dec, 2016.)

As acting director, Woodcock is moving to implement policy and structural changes for OND, including re-evaluating the division-by-division staffing model to determine whether staff can be trained to move between review divisions as needs arise. (Also see “CDER Director Woodcock Plans Changes To Drug Reviews During OND Transition” – Pink Sheet, 6 Mar, 2017.)

Woodcock also has cited the need for more rapid policy development, including guidance documents, to ensure that product sponsors receive the most up-to-date advice. (Also see “Woodcock Wants Rapid Regulatory Policy Development” – Pink Sheet, 22 May, 2017.)

As part of the OND revamp, “we want to develop more time for reviewers to work on specific guidance documents that will help address certain aspects of the review process where we think that the science has evolved to a point where we want to create more modern standards,” Gottlieb said.

The disease-specific guidance writing effort “is an attempt to go back and look at diseases where there haven’t been a lot of innovation in different aspects of clinical development, where we haven’t articulated principles,” Gottlieb said, adding that the forthcoming documents may address issues such as use of different trial designs and endpoints.

Dual Endpoints Under Consideration

At the RAPS meeting, Gottlieb announced that one guidance would address amyotrophic lateral sclerosis. At the FOCR/Alexandria meeting, he revealed Alzheimer’s disease as the focus of another document, adding in jest that “I’ll dribble these out one at a time” in future speeches.

The only Alzheimer’s-specific guidance currently on the books at FDA is a February 2013 draft on developing drugs for the treatment of early-stage disease.

The new Alzheimer’s guidance will “look at some of the new modalities and points that could become maybe not a primary endpoint, but perhaps secondary and tertiary endpoints, or how you look at cognitive function versus performance as different endpoints,” Gottlieb said. “Those are now dual endpoints in a lot of clinical studies.”

With the guidance writing effort, “there’s going to be some things that we’re going to update because the science has changed. In most cases, we’re doing these things anyway now in terms of how we’re thinking about these different therapeutic areas, but we just haven’t had the opportunity to articulate the standards in guidance, and that’s what this exercise is for.”

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